Practical Guidance for Implementing Predictive Biomarkers into Early Phase Clinical Studies

Table 1

Definitions.

Clinical Trial Assay (CTA): a predictive biomarker assay that is either: (1) a prototype form of a planned IVD kit, or (2) a laboratory-developed test that will not be commercialized and sold as a kit to other labs. If the CTA is essential for safe and effective use of the drug, then it must be bridged to a companion diagnostic.
Laboratory-Developed Test (LDT): an in vitro diagnostic test that is developed, validated and used exclusively for in-house diagnostic purposes.
In vitro diagnostic (IVD): any “device” intended for use in the diagnosis of disease or other condition, or in the cure, mitigation, treatment, or prevention of disease, in man or other animals. These devices must be cleared by the FDA through either the 510 (k) premarket notification process or must be approved through the PreMarket Approval (PMA) processes [17].
Companion Diagnostic: an in vitro diagnostic device that provides information that is essential for the safe and effective use of a corresponding therapeutic product [17] and that will be commercialized along with the therapeutic. In general, this test must be clinically validated along with the drug in the registrational trials.
Investigational Use Only (IUO): a regulatory term for a medical device undergoing validation in a clinical trial. A companion diagnostic is labeled as IUO while used in a registrational clinical trial [15].